Key clinical point: Nusinersen provides long-term beneficial changes to the disease course in children with spinal muscular atrophy who initiated treatment when they were presymptomatic.
Major finding: About 84% of study participants have achieved a maximum score on the CHOP INTEND scale.
Study details: An interim analysis of data for 25 infants with spinal muscular atrophy enrolled in the ongoing open-label NURTURE study.
Disclosures: Biogen is sponsoring the study.
Chin R et al. CNS-ICNA 2020, Abstract PL78.